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  1. Policymakers Act on Rare Disease Community’s Behalf

    ... 16, Congress finalized the reauthorization of a bill on stem cells sponsored by U.S. Rep. Chris Smith (R-NJ), who also introduced the ...

    Page last updated 01/08/2016 - 2:39pm.

  2. Therapies

    ... can cause your bone marrow to make more red blood cells. This can improve anemia . Androgens are sometimes used to treat ... from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. ...

    Page last updated 03/18/2016 - 11:26am.

  3. MEDALIST Trial

    ... requirement of ≥ 2 units/8 weeks of packed red blood cells (pRBCs) confirmed for a minimum of 16 weeks immediately preceding ... ≤ 15 μg/L  Prior allogeneic or autologous stem cell transplant Known history of diagnosis of Acute myeloid ...

    Clinical Trial last updated 02/22/2017 - 11:42am.

  4. New therapeutics for myelodysplastic syndromes

    ... MDS is characterized by impaired bone marrow stem cell function, resulting in low blood counts, increased rates of infection ... well as to imbalances among the surrounding, non-cancerous cells (the “MDS microenvironment”).  In this model, progression of an ...

    Research Review last updated 05/02/2016 - 9:28am.

  5. Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

    ... understood, but incomplete elimination of auto-reactive T cells and insufficient stem cell reserve are both possible. Furthermore, 10-15% of SAA patients ...

    Clinical Trial last updated 04/25/2016 - 2:47pm.

  6. What are the treatments for PNH?

    ... This reduces hemolysis , the destruction of red blood cells. Bone marrow/stem cell transplantation (BMT/SCT):  A procedure that replace your unhealthy ...

    FAQ last updated 03/15/2016 - 11:32pm.

  7. Pregnancy in Bone Marrow Failure Disease

    ... without them. But in people who have had an allogeneic stem cell/ bone marrow transplant , the immunosuppressive medications may ... New York University, specializing in the conditions of red cells, platelets and hematologic malignancies. She has been engaged in ...

    Interview last updated 01/13/2017 - 9:28am.

  8. Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT (RIC HSCT NMD)

    ... bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that ... pre-cryopreservation total nucleated cell dose of > 3 x 107 cells/kg, or double unit grafts, each cord blood unit delivering at least ...

    Clinical Trial last updated 06/13/2016 - 2:18pm.

  9. Nonmyeloablative Haploidentical Transplant Followed by MLN9708

    ... post-transplant with MLN9708 to both sensitize tumor cells to NK cytotoxicity and protect against graft-versus-host disease ... reconstitution following haploidentical peripheral blood stem cell transplantation. Patients will receive a nonmyeloablative ...

    Clinical Trial last updated 04/29/2016 - 1:05pm.

  10. Lisa Minter, MD

    ... aplastic anemia , results in the destruction of blood stem and progenitor cells in the bone marrow. One subset of these cells, CD4 T cells, requires the ...

    Grant Recipient last updated 08/18/2014 - 10:07am.